A decision-analysis tool for benefit-risk assessment of nonprescription drugs.

A decision analysis tool is proposed for regulatory assessment of nonprescription drugs. The tool is based on evaluation of each of the drug's potential benefit and risk attributes identified using a value-tree framework. The decision tool is designed as two-factor, two-stage instrument. Each attribute is independently assessed based on both the frequency of occurrence and clinical impact of the attribute. Frequency and clinical impact are scored on a 0-3 scale where a 0 score means no occurrence or no clinical impact, and a 3 means high frequency of occurrence or high clinical impact. The tool is initially used early in drug development to facilitate communication amongst stakeholders and to identify important data gaps. After new data are generated during the development program the tool is used again across the same attributes to yield a final benefit-risk assessment. In both the early assessment and subsequent evaluation use of the Group-Delphi technique and a benefit-risk trade-off heuristic may yield more consistent and coherent outputs. The tool allows regulators to maintain flexibility with respect to how final decisions are made, yet allows specificity and transparency during the evaluation. Further modifications can be incorporated to address drug-specific requirements or regulatory preferences.